Being part of a clinical trial is the most common way that patients receive investigational drugs (i.e. drugs that have not yet been “FDA-approved”). The term “compassionate use” or “compassionate exemption” means that a patient is allowed to receive a drug even though he/she does not meet the eligibility criteria of a clinical trial in which a drug is being studied.

The decision to provide a drug in this manner is made on a case-by-case basis and there must be a reasonable expectation the drug will prolong life or improve a person’s quality of life. In addition, the sponsor of the clinical trial must agree to make the drug available and, as noted in a National Cancer Institute fact sheet, Access to Investigational Drugs, the drug being studied must also meet the following criteria:

There must be substantial clinical evidence that the drug may benefit persons with particular type of cancer.

The drug must be able to be given safely outside a clinical trial.

The drug must be in sufficient supply for ongoing and planned clinical trials.